STOCK TITAN
  1. Home
  2. News
  3. REGMY
  4. RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis

RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis

Rhea-AI Impact
(Neutral)
Rhea-AI Sentiment
(Very Positive)
Tags
clinical trial
Rhea-AI Summary
RemeGen announced that telitacicept (RC18) received Orphan Drug Designation (ODD) from the European Medicines Agency for treating Myasthenia Gravis (MG), becoming the first dual-target biologic drug for MG with ODD from both FDA and EMA globally. The designation provides benefits including protocol assistance, fee reductions, and 10 years of market exclusivity. Telitacicept, already approved in China in May 2025, showed impressive Phase III results with 98.1% of participants showing ≥3 points improvement in MG-ADL (vs 12% placebo) and 87% showing ≥5 points improvement in QMG score (vs 16% placebo). The drug works by blocking BLyS and APRIL signaling pathways to inhibit abnormal B cells and reduce pathogenic autoantibodies. MG affects 15-25 per 100,000 people globally, and current treatments often face challenges with poor response and disease relapse.
RemeGen ha annunciato che telitacicicept (RC18) ha ottenuto la Designazione di Farmaco Ospedaliero Orfano (ODD) dall'Agenzia Europea per i Medicinali per il trattamento della Miastenia Gravis (MG), diventando il primo farmaco biologico a doppio bersaglio per MG ad aver ricevuto l'ODD sia dalla FDA che dall'EMA a livello globale. Questa designazione offre vantaggi quali assistenza nella definizione del protocollo, riduzioni delle tariffe e 10 anni di esclusività di mercato. Telitacicicept, già approvato in Cina nel maggio 2025, ha mostrato risultati impressionanti nella Fase III con il 98,1% dei partecipanti che ha riportato un miglioramento di ≥3 punti nel MG-ADL (contro il 12% del placebo) e l'87% con un miglioramento di ≥5 punti nel punteggio QMG (contro il 16% del placebo). Il farmaco agisce bloccando le vie di segnalazione BLyS e APRIL per inibire le cellule B anomale e ridurre gli autoanticorpi patogeni. La MG colpisce 15-25 persone ogni 100.000 a livello globale e i trattamenti attuali spesso incontrano difficoltà dovute a scarsa risposta e recidive della malattia.
RemeGen anunció que telitacicicept (RC18) recibió la Designación de Medicamento Huérfano (ODD) por parte de la Agencia Europea de Medicamentos para el tratamiento de la Miastenia Gravis (MG), convirtiéndose en el primer medicamento biológico de doble objetivo para MG en obtener la ODD tanto de la FDA como de la EMA a nivel mundial. Esta designación ofrece beneficios como asistencia en el protocolo, reducción de tarifas y 10 años de exclusividad en el mercado. Telitacicicept, ya aprobado en China en mayo de 2025, mostró resultados impresionantes en la Fase III, con un 98,1% de los participantes que presentaron una mejora de ≥3 puntos en MG-ADL (frente al 12% del placebo) y un 87% que mostró una mejora de ≥5 puntos en la puntuación QMG (frente al 16% del placebo). El medicamento actúa bloqueando las vías de señalización BLyS y APRIL para inhibir las células B anormales y reducir los autoanticuerpos patógenos. La MG afecta a 15-25 personas por cada 100.000 en todo el mundo, y los tratamientos actuales a menudo enfrentan desafíos debido a una respuesta pobre y recaídas de la enfermedad.
RemeGen은 텔리타시셉트(RC18)가 유럽의약품청(EMA)으로부터 중증근무력증(MG) 치료를 위한 희귀의약품 지정(ODD)을 받았다고 발표했으며, 이는 전 세계적으로 FDA와 EMA 양쪽에서 ODD를 받은 MG용 최초의 이중 표적 생물학적 제제가 되었습니다. 이 지정은 프로토콜 지원, 수수료 감면, 10년간 시장 독점권 등 혜택을 제공합니다. 텔리타시셉트는 2025년 5월 중국에서 이미 승인되었으며, 3상 임상에서 참가자의 98.1%가 MG-ADL 점수에서 3점 이상 개선(플라세보군 12% 대비), 87%가 QMG 점수에서 5점 이상 개선(플라세보군 16% 대비)을 보였습니다. 이 약물은 BLyS와 APRIL 신호 경로를 차단하여 비정상 B세포를 억제하고 병원성 자가항체를 감소시킵니다. MG는 전 세계적으로 인구 10만 명당 15~25명이 영향을 받으며, 현재 치료법은 반응이 좋지 않거나 질병 재발 문제를 자주 겪고 있습니다.
RemeGen a annoncé que le telitacicicept (RC18) a reçu la désignation de médicament orphelin (ODD) de l'Agence européenne des médicaments pour le traitement de la myasthénie grave (MG), devenant ainsi le premier médicament biologique à double cible pour la MG à obtenir l'ODD à la fois de la FDA et de l'EMA au niveau mondial. Cette désignation offre des avantages tels qu'une assistance au protocole, des réductions de frais et 10 ans d'exclusivité sur le marché. Le telitacicicept, déjà approuvé en Chine en mai 2025, a montré des résultats impressionnants en phase III avec 98,1 % des participants affichant une amélioration de ≥3 points au MG-ADL (contre 12 % pour le placebo) et 87 % une amélioration de ≥5 points au score QMG (contre 16 % pour le placebo). Le médicament agit en bloquant les voies de signalisation BLyS et APRIL pour inhiber les cellules B anormales et réduire les auto-anticorps pathogènes. La MG touche 15 à 25 personnes sur 100 000 dans le monde, et les traitements actuels rencontrent souvent des difficultés liées à une mauvaise réponse et à des rechutes de la maladie.
RemeGen gab bekannt, dass Telitacicicept (RC18) von der Europäischen Arzneimittel-Agentur die Orphan-Drug-Designation (ODD) für die Behandlung der Myasthenia Gravis (MG) erhalten hat und damit das weltweit erste dual-target Biologikum für MG ist, das sowohl von der FDA als auch von der EMA die ODD erhalten hat. Diese Designation bietet Vorteile wie Protokollhilfe, Gebührenreduzierungen und 10 Jahre Marktexklusivität. Telitacicicept, bereits im Mai 2025 in China zugelassen, zeigte beeindruckende Phase-III-Ergebnisse mit 98,1 % der Teilnehmer, die eine Verbesserung von ≥3 Punkten im MG-ADL aufwiesen (vs. 12 % Placebo) und 87 % mit einer Verbesserung von ≥5 Punkten im QMG-Score (vs. 16 % Placebo). Das Medikament wirkt durch Blockierung der BLyS- und APRIL-Signalwege, um abnormale B-Zellen zu hemmen und pathogene Autoantikörper zu reduzieren. MG betrifft weltweit 15-25 von 100.000 Menschen, und aktuelle Behandlungen stoßen häufig auf Probleme mit schlechter Ansprechrate und Krankheitsrückfällen.
Positive
  • First dual-target biologic drug for MG to receive ODD from both FDA and EMA worldwide
  • Impressive Phase III results with 98.1% efficacy vs 12% placebo in MG-ADL improvement
  • Secured 10 years of market exclusivity in Europe through ODD designation
  • Already approved for marketing in China as of May 2025
Negative
  • None.

YANTAI, China, June 17, 2025 /PRNewswire/ -- On June 16, 2025, RemeGen Co., Ltd. ("RemeGen", stock symbols: 688331.SH/09995.HK) announced that telitacicept (RC18; brand name: 泰爱®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for the treatment of Myasthenia Gravis (MG), marking a key milestone achieved in its global development. Telitacicept now is the first dual-target biologic drug for MG with ODD from both FDA and EMA worldwide.

The designation, granted based on telitacicept's significant benefits in treating the life-threatening rare disease of MG, will provide advantages to this novel agent on protocol assistance, regulatory fee reductions/waivers and up to 10 years of market exclusivity, thereby accelerating the clinical development, registration and drug approval process in Europe and its availability among MG patients.

MG is an acquired antibody-mediated rare autoimmune disorder affecting the neuromuscular junction. According to the Myasthenia Gravis Foundation of America (MGFA) and various studies, its global prevalence is estimated to be 15-25 per 100,000, meeting EMA's definition of the rare disease (the prevalence of less than 5 in 10,000 in the EU). Though the current treatment options (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents) may help to manage symptoms, many patients still suffer from poor response, drug intolerance and disease relapse, highlighting a significant unmet clinical need.

Telitacicept is the world's first approved innovative BLyS/APRIL dual-targeting fusion protein drug for MG. The pathology of MG is characterized by the autoantibodies produced by pathological B cells attacking the neuromuscular junction protein (such as acetylcholine receptor, muscle-specific tyrosine kinase). Telitacicept can block both BLyS and APRIL signaling pathways, effectively inhibiting abnormally activated B cells and reducing the production of pathogenic autoantibodies, which is expected to interfere with the source of disease progress of MG.

Telitacicept was approved for marketing in China in May this year. Its phase III clinical trial boasts excellent results: after treatment with telitacicept for 24 weeks, improvement ≥ 3 points in myasthenia gravis-activities of daily living (MG-ADL) was observed in 98.1% of participants (12.0% in the placebo group) and improvements ≥ 5 points in quantitative myasthenia gravis (QMG) score was observed in 87% of participants (16.0% in the placebo group), with statistically significant difference in efficacy from the placebo group and manageable safety profile.

The ODD granted by EMA represents the recognition of telitacicept's innovative mechanism of action and potential in treating MG. RemeGen is proceeding the global multi-center phase III clinical trial of telitacicept in patients with MG to bring the breakthrough treatment option to more patients globally.

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/remegens-telitacicept-rc18-received-orphan-drug-designation-from-ema-for-myasthenia-gravis-302484029.html

SOURCE RemeGen Co., Ltd

FAQ

What is the significance of REGMY's telitacicept receiving EMA Orphan Drug Designation?

The designation provides RemeGen's telitacicept with protocol assistance, fee reductions, and 10 years of market exclusivity in Europe, accelerating development and approval processes for treating Myasthenia Gravis.

What were the Phase III clinical trial results for telitacicept in treating Myasthenia Gravis?

The trial showed 98.1% of participants achieved ≥3 points improvement in MG-ADL (vs 12% placebo) and 87% showed ≥5 points improvement in QMG score (vs 16% placebo), with manageable safety.

How does RemeGen's telitacicept work in treating Myasthenia Gravis?

Telitacicept blocks both BLyS and APRIL signaling pathways, inhibiting abnormally activated B cells and reducing pathogenic autoantibodies that attack neuromuscular junction proteins.

What is the current market status of REGMY's telitacicept?

Telitacicept is currently approved in China (May 2025) and is undergoing global multi-center phase III clinical trials for international expansion.

How common is Myasthenia Gravis, the condition that telitacicept treats?

Myasthenia Gravis affects 15-25 people per 100,000 globally, qualifying as a rare disease under EMA's definition of less than 5 in 10,000 in the EU.
Stock REGMY logo
Remegen

OTC:REGMY

REGMY Rankings

N/A Ranked by Market Cap
N/A Ranked by Dividends

REGMY Latest News

Jun 2, 2025
2025 ASCO ｜Oral Presentation: Disitamab Vedotin Achieves Stellar Efficacy as First-Line Therapy for HER2-Expressing Locally Advanced or Metastatic Gastric Cancer
May 27, 2025
RemeGen: Telitacicept for the Treatment of Generalized Myasthenia Gravis Approved in China
May 12, 2025
Poised to Reshape Treatment Landscape: The Phase 3 Clinical Trial of Disitamab Vedotin as a First-Line Therapy for HER2-Expressing Locally Advanced or Metastatic ‌Urothelial Carcinoma Reached its Primary Endpoints of PFS and OS
Apr 8, 2025
Remegen Announces Exciting Results of Telitacicept Phase 3 Clinical Trial for Patients with Generalized Myasthenia Gravis
Feb 14, 2025
ASCO GU｜RemeGen Announced Highly Encouraging Data from the Phase II Clinical Trial Evaluating Disitamab Vedotin plus Immunotherapy as Perioperative Regimen for Bladder Cancer

REGMY Stock Data

Research and Development in Biotechnology
Professional, Scientific, and Technical Services
China (Mainland)

Related Articles

Continue reading with these related stories

View All Latest News